%0 Journal Article %A Schlenk, Richard %A Lübbert, Michael %A Benner, Axel %A Lamparter, Alexander %A Krauter, Jürgen %A Herr, Wolfgang %A Martin, Hans %A Salih, Helmut R %A Kündgen, Andrea %A Horst, Heinz-A %A Brossart, Peter %A Götze, Katharina %A Nachbaur, David %A Wattad, Mohammed %A Köhne, Claus-Henning %A Fiedler, Walter %A Bentz, Martin %A Wulf, Gerald %A Held, Gerhard %A Hertenstein, Bernd %A Salwender, Hans %A Gaidzik, Verena I %A Schlegelberger, Brigitte %A Weber, Daniela %A Döhner, Konstanze %A Ganser, Arnold %A Döhner, Hartmut %T All-trans retinoic acid as adjunct to intensive treatment in younger adult patients with acute myeloid leukemia: results of the randomized AMLSG 07-04 study. %J Annals of hematology %V 95 %N 12 %@ 0939-5555 %C Berlin %I Springer61936 %M DKFZ-2017-05570 %P 1931-1942 %D 2016 %X The aim of this clinical trial was to evaluate the impact of all-trans retinoic acid (ATRA) in combination with chemotherapy and to assess the NPM1 status as biomarker for ATRA therapy in younger adult patients (18-60 years) with acute myeloid leukemia (AML). Patients were randomized for intensive chemotherapy with or without open-label ATRA (45 mg/m(2), days 6-8; 15 mg/m(2), days 9-21). Two cycles of induction therapy were followed by risk-adapted consolidation with high-dose cytarabine or allogeneic hematopoietic cell transplantation. Due to the open label character of the study, analysis was performed on an intention-to-treat (ITT) and a per-protocol (PP) basis. One thousand one hundred patients were randomized (556, STANDARD; 544, ATRA) with 38 patients treated vice versa. Median follow-up for survival was 5.2 years. ITT analyses revealed no difference between ATRA and STANDARD for the total cohort and for the subset of NPM1-mutated AML with respect to event-free (EFS; p = 0.93, p = 0.17) and overall survival (OS; p = 0.24 and p = 0.32, respectively). Pre-specified PP analyses revealed better EFS in NPM1-mutated AML (p = 0.05) and better OS in the total cohort (p = 0.03). Explorative subgroup analyses on an ITT basis revealed better OS (p = 0.05) in ATRA for genetic low-risk patients according to ELN recommendations. The clinical trial is registered at clinicaltrialsregister.eu (EudraCT Number: 2004-004321-95). %K Tretinoin (NLM Chemicals) %F PUB:(DE-HGF)16 %9 Journal Article %$ pmid:27696203 %2 pmc:PMC5093206 %R 10.1007/s00277-016-2810-z %U https://inrepo02.dkfz.de/record/130491